One-Time gene shot aims to tame rare metabolic disorder
NCT ID NCT06332807
First seen Dec 18, 2025 · Last updated May 22, 2026 · Updated 18 times
Summary
This early-stage trial tests a single dose of NGGT002, a gene therapy that delivers a working copy of the PAH gene, in 12 adults with classic PKU. The goal is to see if it safely lowers harmful phenylalanine levels in the blood. Participants will be monitored for 5 years to check long-term effects.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Atlantic Health System
RECRUITINGMorristown, New Jersey, 07960, United States
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Children's Hospital of Orange County Hospital
RECRUITINGOrange, California, 92868, United States
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55454, United States
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University of Pittsburgh Medical Center
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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University or Texas, Southwestern medical Center
RECRUITINGDallas, Texas, 75390, United States
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Conditions
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